What are stem cells? Stem cells are the body's raw materials — cells from which all other cells with specialized functions are generated. Under the right conditions in the body or a laboratory, stem cells divide to form more cells called daughter cells.

These daughter cells either become new stem cells (self-renewal) or become specialized cells (differentiation) with a more specific function, such as blood cells, brain cells, heart muscle or bone. No other cell in the body has the natural ability to generate new cell types.Stem cells have the remarkable potential to develop into many different cell types in the body during early life and growth. In addition, in many tissues they serve as a sort of internal repair system, dividing essentially without limit to replenish other cells as long as the person or animal is still alive. When a stem cell divides, each new cell has the potential either to remain a stem cell or become another type of cell with a more specialized function, such as a muscle cell, a red blood cell, or a brain cell.

Stem cells are distinguished from other cell types by two important characteristics. First, they are unspecialized cells capable of renewing themselves through cell division, sometimes after long periods of inactivity. Second, under certain physiologic or experimental conditions, they can be induced to become tissue- or organ-specific cells with special functions. In some organs, such as the gut and bone marrow, stem cells regularly divide to repair and replace worn out or damaged tissues. In other organs, however, such as the pancreas and the heart, stem cells only divide under special conditions.

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Scientists have for the first time used adult human stem cells to “cure” rats with Parkinson’s disease, a neurodegenerative illness that currently has no cure. The study, published in the current issue of STEM CELLS Translational Medicine, details how a team of researchers working in Germany at the University of Bielefeld (UB) and Dresden University of Technology were able to produce mature neurons using inferior turbinate stem cells (ITSCs).

ITSCs are stem cells taken from tissue that would generally be discarded after an adult patient undergoes sinus surgery.

The team then tested how the ITSCs would behave when transplanted into a group of rats with Parkinson’s disease. Prior to transplantation, the animals showed severe motor and behavioral deficiencies. However, 12 weeks after receiving the ITSCs, the cells had migrated into the animals’ brains and functional ability was not only fully restored, but significant behavioral recovery was witnessed, too. In another positive sign, no tumors were found in any of the animals after the transplantations, something that also has been a concern in stem cell therapy.Due to their easy accessibility and the resulting possibility of an autologous transplantation approach, ITSCs represent a promising cell source for regenerative medicine,” said UB’s Barbara Kaltschmidt, Ph.D., who led the study along with Alexander Storch, M.D., and Christiana Ossig, M.D., both of Dresden University. “The lack of ethical concerns associated with human embryonic stem cells is a plus, too.”

“In contrast to fighting the symptoms of Parkinson’s disease with medications and devices, this research is focused on restoring the dopamine-producing brain cells that are lost during the disease,” said Anthony Atala, M.D., Editor-in-Chief of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine. "These cells are easy to access and isolate from nasal tissue, even in older patients, which adds to their attraction as a potential therapeutic tool.”

What are stem cells?Stem cells are a renewable source of tissue that can be coaxed to become different cell types of the body. The best-known examples are the embryonic stem (ES) cells found within an early-stage embryo. These cells can generate all the major cell types of the body (they are “pluripotent”). Stem cells have also been isolated from various other tissues, including bone marrow, muscle, heart, gut and even the brain. These “adult” stem cells help with maintenance and repair by becoming specialized cells types of the tissue or organ where they originate. For example, special stem cells in the bone marrow give rise to all the various types of blood cells (similar blood cell-forming stem cells have also been isolated from umbilical cord blood).

Adult vs. embryonic stem cells Because adult stem cells become more committed to a particular tissue type during development, unlike embryonic stem cells, they appear to only develop into a limited number of cell types (they are “multipotent”).

What are induced pluripotent stem cells? In addition to ES cells, induced pluripotent stem (iPS) cells, discovered in 2007, represent an important development in stem cell research to treat diseases like Parkinson’s disease. Essentially, iPS cells are ”man-made” stem cells that share ES cells' ability to become other cell types. IPS cells are created when scientists convert or "reprogram" a mature cell, such as a skin cell, into an embryonic-like state. These cells may have potential both for cell replacement treatment approaches in patients and as disease models that scientists could use in screening new drugs.

IPS cell technology is somewhat related to a previous method called somatic cell nuclear transfer (SCNT) or “therapeutic cloning” (the technology that gave us Dolly the Sheep). Unlike the iPS cell approach, which converts adult cells directly into stem cells, SCNT involves transferring the genetic material of an adult cell into an unfertilized human egg cell, allowing the egg cell to form an early-stage embryo and then collecting its ES cells (which are now genetic “clones” of the person who donated the adult cell). To date, however, this has not been successfully demonstrated with human cells and iPS cell methods may be replacing SCNT as a more viable option.

A potentially exciting use for iPS cells is the development of cell models of Parkinson’s disease. In theory, scientists could use cells from people living with Parkinson’s disease to create iPS cell models of the disease that have the same intrinsic cellular machinery of a Parkinson’s patient. Researchers could use these cell models to evaluate genetic and environmental factors implicated in Parkinson’s disease.Stem cells: What they are and what they do. Stem cells and derived products offer great promise for new medical treatments. Learn about stem cell types, current and possible uses, ethical issues, and the state of research and practice.

By Mayo Clinic Staff

You've heard about stem cells in the news, and perhaps you've wondered if they might help you or a loved one with a serious disease. You may wonder what stem cells are, how they're being used to treat disease and injury, and why they're the subject of such vigorous debate.

Here are some answers to frequently asked questions about stem cells.

﻿Why is there such an interest in stem cells?﻿Researchers and doctors hope stem cell studies can help to:

Increase understanding of how diseases occur. By watching stem cells mature into cells in bones, heart muscle, nerves, and other organs and tissue, researchers and doctors may better understand how diseases and conditions develop.

Generate healthy cells to replace diseased cells (regenerative medicine). Stem cells can be guided into becoming specific cells that can be used to regenerate and repair diseased or damaged tissues in people.

Stem cells may have the potential to be grown to become new tissue for use in transplant and regenerative medicine. Researchers continue to advance the knowledge on stem cells and their applications in transplant and regenerative medicine.

Test new drugs for safety and effectiveness. Before using new drugs in people, some types of stem cells are useful to test the safety and quality of investigational drugs. This type of testing will most likely first have a direct impact on drug development for cardiac toxicity testing.

New areas of study include the effectiveness of using human stem cells that have been programmed into tissue-specific cells to test new drugs. For testing of new drugs to be accurate, the cells must be programmed to acquire properties of the type of cells to be tested. Techniques to program cells into specific cells continue to be studied.

For instance, nerve cells could be generated to test a new drug for a nerve disease. Tests could show whether the new drug had any effect on the cells and whether the cells were harmed.

What are stem cells?Stem cells are the body's raw materials — cells from which all other cells with specialized functions are generated. Under the right conditions in the body or a laboratory, stem cells divide to form more cells called daughter cells.

These daughter cells either become new stem cells (self-renewal) or become specialized cells (differentiation) with a more specific function, such as blood cells, brain cells, heart muscle or bone. No other cell in the body has the natural ability to generate new cell types.

Where do stem cells come from?Researchers have discovered several sources of stem cells:

Embryonic stem cells. These stem cells come from embryos that are three to five days old. At this stage, an embryo is called a blastocyst and has about 150 cells.

These are pluripotent (ploo-RIP-uh-tunt) stem cells, meaning they can divide into more stem cells or can become any type of cell in the body. This versatility allows embryonic stem cells to be used to regenerate or repair diseased tissue and organs, although their use in people has been to date limited to eye-related disorders such as macular degeneration.

Adult stem cells. These stem cells are found in small numbers in most adult tissues, such as bone marrow or fat. Compared with embryonic stem cells, adult stem cells have a more limited ability to give rise to various cells of the body.

Until recently, researchers thought adult stem cells could create only similar types of cells. For instance, researchers thought that stem cells residing in the bone marrow could give rise only to blood cells.

However, emerging evidence suggests that adult stem cells may be able to create unrelated types of cells. For instance, bone marrow stem cells may be able to create bone or heart muscle cells. This research has led to early-stage clinical trials to test usefulness and safety in people. For example, adult stem cells are currently being tested in people with neurological or heart disease.

Adult cells altered to have properties of embryonic stem cells (induced pluripotent stem cells). Scientists have successfully transformed regular adult cells into stem cells using genetic reprogramming. By altering the genes in the adult cells, researchers can reprogram the cells to act similarly to embryonic stem cells.This new technique may allow researchers to use these reprogrammed cells instead of embryonic stem cells and prevent immune system rejection of the new stem cells. However, scientists don't yet know if altering adult cells will cause adverse effects in humans.

Researchers have been able to take regular connective tissue cells and reprogram them to become functional heart cells. In studies, animals with heart failure that were injected with new heart cells experienced improved heart function and survival time.

Perinatal stem cells. Researchers have discovered stem cells in amniotic fluid in addition to umbilical cord blood stem cells. These stem cells also have the ability to change into specialized cells.

Amniotic fluid fills the sac that surrounds and protects a developing fetus in the uterus. Researchers have identified stem cells in samples of amniotic fluid drawn from pregnant women during a procedure called amniocentesis, a test conducted to test for abnormalities.

More study of amniotic fluid stem cells is needed to understand their potential.

Why is there a controversy about using embryonic stem cells?Embryonic stem cells are obtained from early-stage embryos — a group of cells that forms when a woman's egg is fertilized with a man's sperm in an in vitro fertilization clinic. Because human embryonic stem cells are extracted from human embryos, several questions and issues have been raised about the ethics of embryonic stem cell research.

The National Institutes of Health created guidelines for human stem cell research in 2009. Guidelines included defining embryonic stem cells and how they may be used in research and donation guidelines for embryonic stem cells. Also, guidelines stated embryonic stem cells may only be used from embryos created by in vitro fertilization when the embryo is no longer needed.

Where do these embryos come from?The embryos being used in embryonic stem cell research come from eggs that were fertilized at in vitro fertilization clinics but never implanted in a woman's uterus. The stem cells are donated with informed consent from donors. The stem cells can live and grow in special solutions in test tubes or petri dishes in laboratories.

Why can't researchers use adult stem cells instead?Although research into adult stem cells is promising, adult stem cells may not be as versatile and durable as are embryonic stem cells. Adult stem cells may not be able to be manipulated to produce all cell types, which limits how adult stem cells can be used to treat diseases.

Adult stem cells also are more likely to contain abnormalities due to environmental hazards, such as toxins, or from errors acquired by the cells during replication. However, researchers have found that adult stem cells are more adaptable than was initially suspected.

What are stem cell lines and why do researchers want to use them?A stem cell line is a group of cells that all descend from a single original stem cell and is grown in a lab. Cells in a stem cell line keep growing but don't differentiate into specialized cells. Ideally, they remain free of genetic defects and continue to create more stem cells. Clusters of cells can be taken from a stem cell line and frozen for storage or shared with other researchers.

What is stem cell therapy (regenerative medicine), and how does it work?Stem cell therapy, also known as regenerative medicine, promotes the reparative response of diseased, dysfunctional or injured tissue using stem cells or their derivatives. It is the next chapter of organ transplantation and uses cells instead of donor organs, which are limited in supply.

Researchers grow stem cells in a lab. These stem cells are manipulated to specialize into specific types of cells, such as heart muscle cells, blood cells or nerve cells.

The specialized cells can then be implanted into a person. For example, if the person has heart disease, the cells could be injected into the heart muscle. The healthy transplanted heart cells could then contribute to repairing defective heart muscle.

Researchers have already shown that adult bone marrow cells guided to become heart-like cells can repair heart tissue in people, and more research is ongoing.

Have stem cells already been used to treat diseases?Yes, doctors have performed stem cell transplants, also known as bone marrow transplants. In stem cell transplants, stem cells replace cells damaged by chemotherapy or disease or as a way for the donor's immune system to fight some types of cancer and blood-related diseases, such as leukemia. These transplants use adult stem cells or umbilical cord blood.

Researchers are testing adult stem cells to treat other conditions, including a number of degenerative diseases such as heart failure.

What are the potential problems with using embryonic stem cells in humans?To be useful in people, researchers must be certain that stem cells will differentiate into the specific cell types desired.

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